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WO2012006241A3 - Methods and compositions for the specific inhibition of androgen receptor by double-stranded rna - Google Patents

Methods and compositions for the specific inhibition of androgen receptor by double-stranded rna Download PDF

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Publication number
WO2012006241A3
WO2012006241A3 PCT/US2011/042810 US2011042810W WO2012006241A3 WO 2012006241 A3 WO2012006241 A3 WO 2012006241A3 US 2011042810 W US2011042810 W US 2011042810W WO 2012006241 A3 WO2012006241 A3 WO 2012006241A3
Authority
WO
WIPO (PCT)
Prior art keywords
compositions
methods
double
stranded rna
androgen receptor
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US2011/042810
Other languages
French (fr)
Other versions
WO2012006241A2 (en
Inventor
Bob Dale Brown
Henryk T. Dudek
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Dicerna Pharmaceuticals Inc
Original Assignee
Dicerna Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Priority to CA2804210A priority Critical patent/CA2804210A1/en
Priority to EP11804228.2A priority patent/EP2591104A4/en
Priority to JP2013518760A priority patent/JP2013534424A/en
Priority to AU2011276363A priority patent/AU2011276363A1/en
Publication of WO2012006241A2 publication Critical patent/WO2012006241A2/en
Publication of WO2012006241A3 publication Critical patent/WO2012006241A3/en
Priority to US13/723,623 priority patent/US8927515B2/en
Anticipated expiration legal-status Critical
Priority to US14/543,257 priority patent/US9732347B2/en
Ceased legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/08—Drugs for disorders of the urinary system of the prostate
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Urology & Nephrology (AREA)
  • Pulmonology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

This invention relates to compounds, compositions, and methods useful for reducing AR target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
PCT/US2011/042810 2010-07-06 2011-07-01 Methods and compositions for the specific inhibition of androgen receptor by double-stranded rna Ceased WO2012006241A2 (en)

Priority Applications (6)

Application Number Priority Date Filing Date Title
CA2804210A CA2804210A1 (en) 2010-07-06 2011-07-01 Methods and compositions for the specific inhibition of androgen receptor by double-stranded rna
EP11804228.2A EP2591104A4 (en) 2010-07-06 2011-07-01 METHODS AND COMPOSITIONS FOR SPECIFIC INHIBITION OF THE ANDROGEN RECEPTOR BY DOUBLE STRANDED RNA
JP2013518760A JP2013534424A (en) 2010-07-06 2011-07-01 Methods and compounds for the specific inhibition of androgen receptor by double-stranded RNA
AU2011276363A AU2011276363A1 (en) 2010-07-06 2011-07-01 Methods and compositions for the specific inhibition of androgen receptor by double-stranded RNA
US13/723,623 US8927515B2 (en) 2010-07-06 2012-12-21 Methods and compositions for the specific inhibition of androgen receptor by double-stranded RNA
US14/543,257 US9732347B2 (en) 2010-07-06 2014-11-17 Methods and compositions for the specific inhibition of androgen receptor by double-stranded RNA

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US36175910P 2010-07-06 2010-07-06
US61/361,759 2010-07-06

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US13/723,623 Continuation US8927515B2 (en) 2010-07-06 2012-12-21 Methods and compositions for the specific inhibition of androgen receptor by double-stranded RNA

Publications (2)

Publication Number Publication Date
WO2012006241A2 WO2012006241A2 (en) 2012-01-12
WO2012006241A3 true WO2012006241A3 (en) 2012-04-19

Family

ID=45441760

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2011/042810 Ceased WO2012006241A2 (en) 2010-07-06 2011-07-01 Methods and compositions for the specific inhibition of androgen receptor by double-stranded rna

Country Status (6)

Country Link
US (2) US8927515B2 (en)
EP (1) EP2591104A4 (en)
JP (1) JP2013534424A (en)
AU (1) AU2011276363A1 (en)
CA (1) CA2804210A1 (en)
WO (1) WO2012006241A2 (en)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100048912A1 (en) 2008-03-14 2010-02-25 Angela Brodie Novel C-17-Heteroaryl Steroidal CYP17 Inhibitors/Antiandrogens, In Vitro Biological Activities, Pharmacokinetics and Antitumor Activity
ES2566750T3 (en) 2008-04-16 2016-04-15 The Johns Hopkins University Method to determine the risk of prostate cancer recurrence
US9175291B2 (en) 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
US9850486B2 (en) 2012-12-14 2017-12-26 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of CKAP5 by double-stranded RNA
EP2968370A4 (en) 2013-03-14 2016-09-21 Univ Maryland ANDROGEN RECEPTOR DECREASING AGENTS AND USES THEREOF
CA2916499A1 (en) 2013-07-03 2015-01-08 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna
BR112016002970A2 (en) 2013-08-12 2017-09-12 Tokai Pharmaceuticals Inc biomarkers for the treatment of neoplastic disorders using androgen-directed therapies
WO2015179404A1 (en) 2014-05-19 2015-11-26 The Johns Hopkins University Methods for identifying androgen receptor splice variants in subjects having castration resistant prostate cancer
CA2959336A1 (en) * 2014-08-25 2016-03-03 The Johns Hopkins University Methods and compositions related to prostate cancer therapeutics
US10676737B2 (en) 2014-12-17 2020-06-09 Proqr Therapeutics Ii B.V. Targeted RNA editing
JP2018527022A (en) * 2015-07-30 2018-09-20 バイエル・クロップサイエンス・アクチェンゲゼルシャフト Method and composition for controlling rust fungi by inhibiting expression of HXT1 gene
MA45340A (en) * 2016-04-01 2019-02-06 Avidity Biosciences Llc ANDROGEN RECEPTOR NUCLEIC ACIDS AND THEIR USES
CA3024944A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
CN110352244B (en) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ Chemically modified RNA editing single stranded oligonucleotides
CN110177544A (en) 2016-11-29 2019-08-27 普尔泰克健康有限公司 Exosomes for delivery of therapeutic agents
EP3571300A1 (en) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
KR102321426B1 (en) * 2017-02-21 2021-11-05 올릭스 주식회사 Asymmetric siRNA Inhibiting Expression of Genes Directed to Male Type Depilation
US20200399681A1 (en) * 2018-02-23 2020-12-24 Cornell University Assay for detection of androgen receptor variants
CA3095311A1 (en) 2018-03-27 2019-10-03 University Of Rochester Nucleic acid molecules for pseudouridylation
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
KR102473989B1 (en) * 2018-11-28 2022-12-07 (주)바이오니아 Double-stranded oligo nucleotide structure comprising androgen receptor specific sequences and composition comprising the same for preventing hair loss and hair growth
WO2025160224A1 (en) * 2024-01-24 2025-07-31 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of androgen receptor (ar), compositions thereof, and methods of use
WO2025226946A1 (en) * 2024-04-24 2025-10-30 Cedric Francois Methods and compositions for the treatment of androgenic alopecia

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050164970A1 (en) * 2003-12-22 2005-07-28 University Of Kansas Medical Center Method for treating prostate cancer using siRNA duplex for androgen receptor
US20050170371A1 (en) * 2001-05-18 2005-08-04 Sirna Therapeutics, Inc. RNA interference mediated inhibition of 5-alpha reductase and androgen receptor gene expression using short interfering nucleic acid (siNA)
US20060257851A1 (en) * 2002-11-26 2006-11-16 Itzhak Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
US20090043083A1 (en) * 2004-03-15 2009-02-12 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded rna

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8090542B2 (en) * 2002-11-14 2012-01-03 Dharmacon Inc. Functional and hyperfunctional siRNA
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
JP4961549B2 (en) * 2006-02-16 2012-06-27 国立大学法人愛媛大学 SiRNA specific for androgen receptor gene
US20080014584A1 (en) * 2006-06-01 2008-01-17 Mayo Foundation For Medical Education And Research Modulating androgen receptor activity

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050170371A1 (en) * 2001-05-18 2005-08-04 Sirna Therapeutics, Inc. RNA interference mediated inhibition of 5-alpha reductase and androgen receptor gene expression using short interfering nucleic acid (siNA)
US20060257851A1 (en) * 2002-11-26 2006-11-16 Itzhak Bentwich Bioinformatically detectable group of novel viral regulatory genes and uses thereof
US20050164970A1 (en) * 2003-12-22 2005-07-28 University Of Kansas Medical Center Method for treating prostate cancer using siRNA duplex for androgen receptor
US20090043083A1 (en) * 2004-03-15 2009-02-12 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded rna

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
See also references of EP2591104A4 *

Also Published As

Publication number Publication date
US20150197756A1 (en) 2015-07-16
AU2011276363A1 (en) 2013-01-10
EP2591104A2 (en) 2013-05-15
CA2804210A1 (en) 2012-01-12
EP2591104A4 (en) 2014-09-03
US8927515B2 (en) 2015-01-06
US9732347B2 (en) 2017-08-15
WO2012006241A2 (en) 2012-01-12
US20130131149A1 (en) 2013-05-23
JP2013534424A (en) 2013-09-05

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